-- The mean endothelial function PAT score improved from the abnormal to

the normal range (>1.67) in SCD patients with an abnormal PAT score at

baseline.

-- Improvement in endothelial dysfunction appeared to be dose-dependent.

-- 6R-BH4 was well-tolerated in sickle cell disease patients.

Pending feedback from the FDA at a pre-IND meeting in November, future
development plans could include a cross-sectional study in sickle cell
disease patients to assess the relationship between endothelial dysfunction
and the frequency and severity of sickle cell crises, and a three month
double-blind, placebo-controlled dose finding study with a primary endpoint
of improvement in endothelial function.

Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin stated, "We
are very encouraged by the study results as it suggests that the known
severe endothelial dysfunction present in sickle cell disease patients is
due to an acquired BH4 deficiency and is responsive to 6R-BH4 replacement
therapy. Endothelial dysfunction in SCD is now believed to play a greater
role in the pathophysiology of sickle cell vasoocclusive crises than had
previously been realized. The ability to reverse this dysfunction with oral
6R-BH4 therapy represents a new opportunity to treat SCD."

Dr. Kakkis continued, "The EndoPAT device is a new reproducible method
to measure the effect of endothelial dysfunction on small vessel function
and has been adopted by the ongoing Framingham study of cardiovascular
risks and outcomes as recently published. The PAT score has been shown to
correlate with other cardiovascular risk factors and endothelial
dysfunction in general has been shown to predict adverse cardiovascular
events in at least 10 prospective studies. With this current SCD study, we
have an indication that 6R-BH4 is active in improving endothelial function,
and we hope to establish a clear relationship between the degree of
endothelial dysfunction and the frequency and severity of sickle cell
crises using a survey of SCD patients with the EndoPAT test."

"Sickle cell disease is a chronic, lifelong disease that affects a
significantly large population of approximately 100,000 people in the U.S.
These patients suffer periods of intense pain, poor blood flow, organ
damage and other serious medical complications. To date, however, there are
very limited treatment options for this debilitating disease and average
life expectancy is only in the forties," said Lewis Hsu, M.D., Ph.D.,
Associate Professor of Pediatric Hematology & Interim Director of Marian
Anderson Comprehensive Sickle Cell Center, St. Christopher's Hospital for
Children, Drexel University College of Medicine, Philadelphia, PA. "These
study findings are significant and support the concept of using BH4 to
treat sickle cell disease, a somewhat novel but plausible approach. Recent
reviews of the pathophysiology in sickle cell disease have shifted the
focus away from the physical sickling of red blood cells and more toward
the deteriorating health of the blood vessels resulting from hemolysis and
the release of hemoglobin from red cells."

Study Design

The Phase 2a multi-center, open-label study enrolled 32 subjects and
was conducted at 12 U.S. sites. Among other eligibility criteria, to
participate in the study, SCD patients were at least 15 years of age and
were not receiving hydroxyurea or hypertransfusion therapy. Study patients
received 6R-BH4 at 2.5 mg/kg, 5 mg/kg, 10 mg/kg and 20 mg/kg for four weeks
each in a 16-week dose-escalation study. A total of 21 patients completed
the baseline and final assessments.

The primary objective in the study was to evaluate the safety of oral
6R-BH4 administered in escalating doses in patients with sickle cell
disease. The secondary objective was to evaluate changes in physiological
and biochemical markers of endothelial function which underlie some key
aspects of SCD. PAT scores were measured at the end of each 4 week dose
period and compared to the baseline PAT score for statistical purposes.

BioMarin will hold a conference call today, October 15, 2008, at 5:00
p.m. ET to discuss the results of the Phase 2a study of 6R-BH4 in sickle
cell disease. This event can be accessed on the investor section of the
BioMarin website at http://www.BMRN.com.

Date: October 15, 2008

Time: 5:00 p.m. ET

U.S. and Canada Toll-Free Dial in #: 866.356.4123

International Dial in #: 617.597.5393

Participant Code: 75782382

Replay Toll-Free Dial in #: 888.286.8010

Replay International Dial in #: 617.801.6888

Replay Code: 73130467

About 6R-BH4

6R-BH4, commonly known as BH4 or tetrahydrobiopterin, is a naturally
occurring enzyme cofactor that is required for numerous biochemical and
physiologic processes, including the synthesis of nitric oxide (NO). NO has
been shown to play a key protective role throughout the cardiovascular
system and produces multiple positive effects, such as relaxing smooth
muscle, reducing blood pressure, controlling inflammation and reducing
platelet aggregation. Researchers have demonstrated that a deficiency of
BH4 can disrupt NO synthesis, resulting in a loss of normal endothelial NO
production. This loss of endothelial NO production, commonly referred to as
endothelial dysfunction, has been associated with many cardiovascular
diseases, including hypertension, diabetic vascular disease, peripheral
arterial disease, coronary arterial disease and pulmonary hypertension, and
has been shown to be a strong predictor of cardiovascular adverse events in
a number of clinical studies.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for
serious diseases and medical conditions. The company's product portfolio
comprises three approved products and multiple clinical and pre-clinical
product candidates. Approved products include Naglazyme(R) (galsulfase) for
mucopolysaccharidosis VI (MPS VI), a product wholly developed and
commercialized by BioMarin; Aldurazyme(R) (laronidase) for
mucopolysaccharidosis I (MPS I), a product which BioMarin developed through
a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin
dihydrochloride) Tablets, a product for the treatment of phenylketonuria
(PKU), developed in partnership with Merck Serono, a division of Merck KGaA
of Darmstadt, Germany. Other product candidates include 6R-BH4 for
cardiovascular indications, which is currently in Phase 2 clinical
development for the treatment of peripheral arterial disease and sickle
cell disease, and PEG-PAL (PEGylated recombinant phenylalanine ammonia
lyase), which is currently in Phase 1 clinical development for the
treatment of PKU. For additional information, please visit
http://www.BMRN.com. Information on BioMarin's website is not incorporated
by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including, without
limitation, statements about: expectations related to BioMarin's clinical
trials of 6R-BH4 for sickle cell disease and other indications, actions by
regulatory authorities and the general development of BH4 for sickle cell.
These forward-looking statements are predictions and involve risks and
uncertainties such that actual results may differ materially from these
statements. These risks and uncertainties include, among others: results
and timing of current and planned preclinical studies and clinical trials;
the ability to enroll patients in future trials in a timely manner; the
actual correlation between PAT scores and clinical endpoints; the content
and timing of decisions by the U.S. Food and Drug Administration, the
European Commission and other regulatory authorities concerning each of the
described products and product candidates; the market for each of these
products; and those factors detailed in BioMarin's filings with the
Securities and Exchange Commission, including, without limitation, the
factors contained under the caption "Risk Factors" in BioMarin's 2007
Annual Report on Form 10-K, and the factors contained in BioMarin's reports
on Form 10-Q. Stockholders are urged not to place undue reliance on
forward-looking statements, which speak only as of the date hereof.
BioMarin is under no obligation, and expressly disclaims any obligation to
update or alter any forward-looking statement, whether as a result of new
information, future events or otherwise.

BioMarin(R) , Naglazyme(R) and Kuvan(R) are a registered trademarks of
BioMarin Pharmaceutical Inc.
Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.

(Date:12/9/2016)... Research and Markets has announced the addition of the "High Throughput ... ... the market by the following Technology Types: Label-Free Technology, Cell-Based Assay, and ... Canada , Japan , Europe ... and Rest of World. Annual estimates and forecasts are ...

(Date:12/9/2016)... Research and Markets has announced the ... 2021" report to their offering. ... Market growth can be ... the pharmaceutical and biotechnology industry and technological advancements like automation ... markets and growing research activities in toxicology and stem cells ...

(Date:12/8/2016)... FL (PRWEB) , ... December 08, 2016 , ... ... awarded accreditation with distinction by the Undersea and Hyperbaric Medical Society (UHMS), the ... with advanced care and patient safety. Only a few hospitals and facilities have ...

(Date:12/8/2016)... ... 08, 2016 , ... Mirixa Corporation , a leading ... pharmacist-delivered patient care services, has announced the promotions of Karen Litsinger to senior ... of sales. , Litsinger joined Mirixa in 2008 after serving as a ...

(Date:12/8/2016)... ... December 08, 2016 , ... SunView Software aims to redefine ... are both engaging and easy to use. Coming off the heels of a ... today its plans to roll out new AI-powered self-service enhancements to help organizations ...